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First Patient Enrolled in Global Phase 3 Clinical Program for Elamipretide in Patients with Dry Age-Related Macular Degeneration

NEEDHAM, Mass., June 05, 2024. Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biopharmaceutical company focused on the discovery, development and commercialization of novel therapies for diseases involving mitochondrial dysfunction, today announced that it has enrolled and dosed its first patient in the ReNEW trial (NCT06373731) as part of its Phase 3 clinical program for elamipretide in patients with dry age-related macular degeneration (dry AMD). The program consists of the two Phase 3 trials, ReNEW and ReGAIN. Both trials will evaluate the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide in participants with dry AMD. The primary efficacy endpoint will measure the rate of change in the macular area of photoreceptor loss, assessed by spectral domain-optical coherence tomography (SD-OCT) and ellipsoid zone (EZ) mapping at week 48.

"Enrolling the first patient in our Phase 3 program puts us one step closer to bringing a potential first-in-class, at-home treatment option to patients living with dry AMD," said Reenie McCarthy, Chief Executive Officer. "With multiple sites now active and able to enroll patients in the U.S., we are off to a great start and look forward to evaluating the efficacy and safety of elamipretide in patients with dry AMD in these pivotal Phase 3 trials."

"There remains a tremendous need for new therapies with differentiated mechanisms of action to treat dry AMD, especially those that could be readily employed for the management of earlier stages of disease," said Charles Wykoff, MD, PhD from Retinal Consultants of Texas. "Elamipretide, a mitochondrial targeted therapy, can be self-administered by patients at home. It holds the potential to be a transformative approach to dry AMD management by targeting the bioenergetic imbalance implicated in the progressive loss of photoreceptors that defines AMD and leads to progressive visual decline."

About ReNEW and ReGAIN
ReNEW and ReGAIN are Phase 3 global clinical trials evaluating the efficacy and safety of once-daily subcutaneous injections of elamipretide in participants with dry AMD. The primary endpoint for the trials is the rate of change in the macular area of photoreceptor loss assessed by spectral domain-optical coherence tomography and ellipsoid zone mapping. In the ReNEW trial, 360 patients will be randomized 2:1 to either elamipretide or placebo for 96 weeks with the option for participants to enroll in the open-label extension trial, ReTAIN.

About dry AMD
AMD is the leading cause of irreversible blindness and is estimated by the Vision and Eye Health Surveillance System to affect 19.8 million Americans aged 40 and older (Rein 2022), with 85% to 90% of cases being dry AMD (Schultz 2021). Dry AMD is a progressive retinal disease in which the photoreceptors, which are specialized neurons found in the retina that convert light into electrical signals required for normal visual function, suffer progressive damage and death, leading to loss of vision. Mitochondrial dysfunction, which has been associated with aging, smoking, obesity, and cardiovascular health, is known to precede clinical symptoms of AMD and increase commensurate with AMD disease progression (Feher 2006; Karunadharma 2010; Terluk 2015). Photoreceptor loss can be quantified by measuring the thickness between the EZ and retinal pigment epithelium (i.e., EZ-RPE thickness). Loss of photoreceptors cells, or EZ attenuation, has been shown to precede and predict the loss of visual function and areas of geographic atrophy in dry AMD.

About Stealth BioTherapeutics
Stealth BioTherapeutics is a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel therapies for age-related and rare genetic diseases involving mitochondrial dysfunction. The Company is initiating Phase 3 clinical trials of elamipretide, its lead investigational product candidate, in dry age-related macular degeneration. Elamipretide is also being tested in a fully enrolled Phase 3 clinical trial in primary mitochondrial myopathy, a rare skeletal myopathic disease, and is under review by the Food and Drug Administration for Barth syndrome, an ultra-rare cardioskeletal disease. The Company is developing its second-generation clinical-stage candidate, bevemipretide (SBT-272), for ophthalmic and neurological disease indications. The Company has a deep pipeline of novel mitochondria-targeted compounds under evaluation as therapeutic product candidates.

SOURCE Stealth BioTherapeutics Inc.

Posted: June 2024

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